Doxycycline Injection for Neurofibromatosis Type 2

Overseen By Christopher Wood

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Massachusetts Eye and Ear Infirmary

Must not be taking: Anticancer therapies, Glucocorticoids

Disqualifiers: Allergy to doxycycline, Unstable disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 4

Duration: 1 day

19 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method to treat skin schwannomas (tumors on nerve tissue) in individuals with neurofibromatosis type 2 (NF2). Researchers are testing the direct injection of doxycycline, a common antibiotic, into the tumors to determine if it can shrink them without surgery. The trial is open to individuals diagnosed with NF2 who have specific measurable skin tumors, excluding those on the face. Participants must not be allergic to doxycycline and should not be receiving certain other cancer treatments. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this innovative approach.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are currently receiving anticancer therapies or have had them within 4 weeks before the study. It's best to discuss your specific medications with the study team.

Is there any evidence suggesting that doxycycline injection is likely to be safe for humans?

Research has shown that doxycycline, an antibiotic, is generally safe for humans. However, specific safety information is lacking for its use as an injection to treat skin schwannomas (a type of non-cancerous tumor) related to neurofibromatosis type 2 (NF2). As this trial is in its early stages, the main goal is to assess treatment tolerance and identify any possible side effects.

Doxycycline has FDA approval for other uses, which provides some reassurance about its safety. However, using it as an injection for this specific condition is new. Participants in this trial will help researchers gather more safety information. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for neurofibromatosis type 2?

Unlike the standard treatments for Neurofibromatosis Type 2, which often include surgery or radiation to manage tumors, the doxycycline injection offers a novel approach. This treatment is unique because it utilizes doxycycline, an antibiotic, in an injectable form to potentially reduce tumor growth. Researchers are excited about this because doxycycline is thought to inhibit an enzyme that tumors need to grow, offering a targeted, non-invasive option. This could provide a less aggressive and more focused treatment compared to existing methods.

What evidence suggests that doxycycline injection might be an effective treatment for neurofibromatosis type 2?

Research has shown that doxycycline might slow tumor growth, making it a promising option for treating conditions like Neurofibromatosis Type 2 (NF2). Studies have found that injecting doxycycline directly into skin tumors, known as schwannomas, might help shrink them. This targeted treatment could reduce the need for surgery, which often carries risks and high costs. Early results suggest that doxycycline not only shrinks tumors but also increases cancer cell death, potentially improving treatment outcomes. If successful, this approach could offer a simpler and safer way to manage NF2-related skin tumors. ^{1 2 3 4 6}

Who Is on the Research Team?

D. Bradley Welling, MD, PhD

Principal Investigator

Massachusetts Eye and Ear Infirmary

Are You a Good Fit for This Trial?

This trial is for individuals aged 8 or older with neurofibromatosis type 2 (NF2) and skin schwannomas. Participants must have stable neurological conditions, a life expectancy over a year, and be able to give consent. They should not be on recent anticancer treatments or have severe medical issues that could affect participation.

Inclusion Criteria

I am 8 years old or older.

I have no lingering side effects from previous cancer treatments.

My neurological symptoms have been stable for at least a week.

Exclusion Criteria

My tumor is on my face or affects major nerves for movement.

I haven't had radiation on the tumor I want to study in the last year or any radiation in the last 4 weeks.

I am not pregnant, breastfeeding, and if capable of having children, I am using effective birth control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive local doxycycline intra-tumoral injection for NF2-related skin schwannomas

1 day

1 visit (in-person)

Follow-up

Participants are monitored for changes in tumor size and other symptoms

6 months

2 visits (in-person)

Long-term Follow-up

Participants are monitored for changes in tumor size and other symptoms

1 year

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Doxycycline Injection

Trial Overview The study tests local injections of Doxycycline directly into the skin tumors of NF2 patients as an alternative to surgery. The goal is to see if this method can effectively reduce tumor size while being less risky and costly than standard surgical procedures.

How Is the Trial Designed?

Group I: Single Arm Open Label Experimental Treatment 1 Intervention

Open Label

Who Is Running the Clinical Trial?

Massachusetts Eye and Ear Infirmary

Lead Sponsor

Trials

115

Recruited

15,000+

Published Research Related to This Trial

Topical photodynamic therapy (PDT) using aminolevulinic acid (ALA) was shown to effectively target cutaneous neurofibromas (cNF) in a phase I trial with 27 participants, demonstrating significant fluorescence in tumors and minimal impact on surrounding normal skin.

The treatment resulted in a notable increase in apoptotic cells in ALA-treated tumors compared to vehicle-treated tumors, indicating that PDT may provide a less invasive and tissue-sparing option for managing neurofibromatosis type 1.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Photodynamic Therapy for Benign Cutaneous Neurofibromas Using Aminolevulinic Acid Topical Application and 633 nm Red Light Illumination. Quirk, B., Olasz, E., Kumar, S., et al.[2021]

Citations

1. rally.massgeneralbrigham.org rally.massgeneralbrigham.org/study/nf2doxy

Do you (or your child) have an NF2-related skin ... We are doing this research to study local doxycycline injection as a potential treatment for NF2-related skin schwannomas, ultimately reducing the risks and ...

2. clinicaltrials.gov clinicaltrials.gov/ct2/show/NCT05521048?term=doxycycline&draw=2&rank=3

Doxycycline in Cutaneous Schwannoma (NF2) Data collected at the beginning of a clinical study for all participants and for each arm or comparison group. These data include demographics, such as age, sex ...

3. withpower.com withpower.com/trial/phase-2-neurofibromatosis-type-2-8-2022-6b1dd

Doxycycline Injection for Neurofibromatosis Type 2 Research shows that doxycycline can enhance the effectiveness of certain cancer treatments by reducing tumor growth and increasing the death of cancer cells.

4. ctv.veeva.com ctv.veeva.com/study/doxycycline-in-cutaneous-schwannoma-nf2

Doxycycline in Cutaneous Schwannoma (NF2) Specifically, local doxycycline intra-tumoral injection will be performed as a potential treatment for NF2-related skin schwannomas, ultimately ...

5. trialx.com trialx.com/clinical-trials/listings/249482/doxycycline-in-cutaneous-schwannoma-nf2/

Doxycycline in Cutaneous Schwannoma (NF2) Aims: To demonstrate change in peripheral schwannoma size following direct injection into the tumor with doxycycline hyclate. Nineteen ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC4926229/

Preclinical Efficacy and Safety Profile of Allometrically Scaled ... Effectiveness and preclinical safety profile of doxycycline to be used “off-label” to induce therapeutic transgene expression in a phase I clinical trial for ...